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A Cost-Effectiveness Analysis of Adjuvant Nivolumab for Patients with Resected Esophageal Cancer or Gastroesophageal Junction Cancer in France. 法国对已切除食管癌或胃食管交界癌患者进行 Nivolumab 辅助治疗的成本效益分析。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-07-04 DOI: 10.1007/s41669-024-00500-y
Paul Casabianca, Marc Massetti, François-Emery Cotte, Romain Moreau, Sarah Kassahun, Prianka Singh, Inkyu Kim, Anne-Françoise Gaudin, Guillaume Piessen, Henri Leleu
{"title":"A Cost-Effectiveness Analysis of Adjuvant Nivolumab for Patients with Resected Esophageal Cancer or Gastroesophageal Junction Cancer in France.","authors":"Paul Casabianca, Marc Massetti, François-Emery Cotte, Romain Moreau, Sarah Kassahun, Prianka Singh, Inkyu Kim, Anne-Françoise Gaudin, Guillaume Piessen, Henri Leleu","doi":"10.1007/s41669-024-00500-y","DOIUrl":"10.1007/s41669-024-00500-y","url":null,"abstract":"<p><strong>Introduction: </strong>Esophageal and gastroesophageal junction cancer (EC/GEJC) is a poor prognosis disease with a high risk of recurrence even in patients curatively resected. Adjuvant nivolumab is currently used for patients with completely resected (R0) EC/GEJC who have residual pathologic disease following prior neoadjuvant chemoradiotherapy. This study aimed to determine the cost effectiveness of nivolumab in this indication in France according to the collective perspective excluding indirect costs.</p><p><strong>Materials and methods: </strong>A simplified four-health-state semi-Markov model was developed to model EC/GEJC patients who have residual disease after neoadjuvant chemoradiotherapy followed by R0 over a 15-year time horizon, comparing adjuvant nivolumab versus surveillance, which was the recommended French clinical practice before immunotherapy arrival. Time-to-recurrence (TTR) from CheckMate 577 was used to inform transition from disease-free to post-recurrence health state; patients who recurred were split according to the distribution of type of recurrence observed during the trial. Post-recurrence survival (PRS) according to the type of recurrence was derived from a real-world registry.</p><p><strong>Results: </strong>Adjuvant treatment with nivolumab led to an incremental survival gain of 1.19 years (+ 34%), mostly in the disease-free state, an incremental cost of €48,634 and QALY of 0.98 resulting in an incremental cost-utility ratio (ICUR) of €49,572/QALY with limited uncertainty. 'Cure assumption' at 5 years had an important impact on the results (€41,115/QALY; - 17%), as that tends to increase life-years and QALYs while costs remain the same. Probabilistic sensitivity analyses confirmed reference ICUR (€52,542/QALY) with 80% probability of nivolumab being cost effective at a willingness-to-pay threshold of €75,000/QALY.</p><p><strong>Conclusions: </strong>Our analysis suggests that adjuvant nivolumab is cost effective in the treatment of EC/GEJC patients who have residual disease after neoadjuvant CRT followed by R0 resection. Compared with previously evaluated cost-effectiveness analyses for other immune-checkpoint inhibitors indicated in metastatic settings, ICUR appears particularly low in this early setting thanks to the important impact on health outcomes and capped treatment duration.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"689-699"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362429/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141535054","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Mapping Payment and Pricing Schemes for Health Innovation: Protocol of a Scoping Literature Review. 绘制卫生创新的支付和定价方案:范围界定文献审查协议》。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-05-21 DOI: 10.1007/s41669-024-00496-5
Vittoria Ardito, Ludovico Cavallaro, Michael Drummond, Oriana Ciani
{"title":"Mapping Payment and Pricing Schemes for Health Innovation: Protocol of a Scoping Literature Review.","authors":"Vittoria Ardito, Ludovico Cavallaro, Michael Drummond, Oriana Ciani","doi":"10.1007/s41669-024-00496-5","DOIUrl":"10.1007/s41669-024-00496-5","url":null,"abstract":"<p><strong>Introduction: </strong>Innovative pricing and payment/reimbursement schemes have been proposed as one part of the solution to the problem of patient access to new health technologies or to the uncertainty about their long-term effectiveness. As part of a Horizon Europe research project on health innovation next generation pricing and payment models (HI-PRIX), this protocol illustrates the conceptual and methodological steps related to a scoping review aiming at investigating nature and scope of pricing and payment/reimbursement schemes applied to, or proposed for, existing or new health technologies.</p><p><strong>Methods: </strong>A scoping review of literature will be performed according to the PRISMA guidelines for scoping reviews (PRISMA-ScR) guidelines. The search will be conducted in three scientific databases (i.e., PubMed, Web of Science, and Scopus), over a 2010-2023 timeframe. The search strategy is structured around two blocks of keywords, namely \"pricing and payment/reimbursement schemes,\" and \"innovativeness\" (of the scheme type or scheme use). A simplified search will be replicated in the gray literature. Studies illustrating pricing and payment/reimbursement schemes with a sufficient level of details to explain their characteristics and functioning will be deemed eligible to be considered for data synthesis. Pricing and payment/reimbursement schemes will be classified according to several criteria, such as their purpose, nature, governance, data collection needs, and foreseen distribution of risk. The results will populate a publicly available online tool, the Pay-for-Innovation Observatory.</p><p><strong>Discussion: </strong>The findings of this review have the potential to offer a comprehensive toolkit with a variety of pricing and payment schemes to policymakers and manufacturers facing reimbursement and access decisions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"765-772"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141076629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Modeling First-Line Daratumumab Use for Newly Diagnosed, Transplant-Ineligible, Multiple Myeloma: A Cost-Effectiveness and Risk Analysis for Healthcare Payers. 为新诊断、不符合移植条件的多发性骨髓瘤一线使用达拉单抗建模:医疗支付方的成本效益和风险分析。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-06-20 DOI: 10.1007/s41669-024-00503-9
Diana Beatriz Bayani, Yihao Clement Lin, Chandramouli Nagarajan, Melissa G Ooi, Allison Ching Yee Tso, John Cairns, Hwee Lin Wee
{"title":"Modeling First-Line Daratumumab Use for Newly Diagnosed, Transplant-Ineligible, Multiple Myeloma: A Cost-Effectiveness and Risk Analysis for Healthcare Payers.","authors":"Diana Beatriz Bayani, Yihao Clement Lin, Chandramouli Nagarajan, Melissa G Ooi, Allison Ching Yee Tso, John Cairns, Hwee Lin Wee","doi":"10.1007/s41669-024-00503-9","DOIUrl":"10.1007/s41669-024-00503-9","url":null,"abstract":"<p><strong>Background and objective: </strong>This study aimed to assess the cost-effectiveness of two regimens regarded as the standard of care for the treatment of newly diagnosed, transplant-ineligible multiple myeloma in Singapore: (1) daratumumab, lenalidomide, and dexamethasone and (2) bortezomib, lenalidomide, and dexamethasone. Additionally, it aimed to explore potential strategies to manage decision uncertainty and mitigate financial risk.</p><p><strong>Methods: </strong>A cost-effectiveness analysis from the healthcare system perspective was conducted using a partitioned survival model to estimate lifetime costs and quality-adjusted life years (QALYs) associated with daratumumab-based treatment and the bortezomib-based regimen. The analysis used data from the MAIA and SWOG S0777 trials and incorporated local real-world data where available. Sensitivity analyses were performed to evaluate the robustness of the findings, and a risk analysis was conducted to analyze various payer strategies in terms of their payer strategy and uncertainty burden (P-SUB), which account for the decision uncertainty and the additional cost of choosing a suboptimal intervention.</p><p><strong>Results: </strong>The incremental cost-effectiveness ratio (ICER) for daratumumab, lenalidomide, and dexamethasone (DRd) compared with bortezomib, lenalidomide, and dexamethasone (VRd) was US $90,364 per QALY gained. The results were sensitive to variations in survival for DRd, postprogression treatment costs, cost of hospice care, and hazard ratio for progression-free survival. The scenarios explored indicated that structural assumptions, such as the time horizon of the analysis, significantly influenced the results due to uncertainties arising from immature trial data and treatment efficacy over time. Among the various payer strategies compared, an upfront price discount for daratumumab emerged as the best approach with the lowest P-SUB at US $14,708.</p><p><strong>Conclusion: </strong>In conclusion, this study finds that daratumumab as a first-line treatment for myeloma exceeds the cost-effectiveness threshold considered in this evaluation. An upfront price reduction is the recommended strategy to manage uncertainties and mitigate financial risks. These findings highlight the importance of targeted payer strategies to address specific types and sources of uncertainty.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"651-664"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362436/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141427407","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Using a Patient-Centered Multicriteria Decision Analysis to Assess the Value of Multiple Sclerosis Treatments in the US: A Study Protocol. 使用以患者为中心的多标准决策分析评估美国多发性硬化症治疗的价值:研究方案。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-07-09 DOI: 10.1007/s41669-024-00509-3
Surachat Ngorsuraches, Tim C Lai, Rebecca Habermann, Yolanda Wheeler, William Meador
{"title":"Using a Patient-Centered Multicriteria Decision Analysis to Assess the Value of Multiple Sclerosis Treatments in the US: A Study Protocol.","authors":"Surachat Ngorsuraches, Tim C Lai, Rebecca Habermann, Yolanda Wheeler, William Meador","doi":"10.1007/s41669-024-00509-3","DOIUrl":"10.1007/s41669-024-00509-3","url":null,"abstract":"<p><strong>Objective: </strong>The engagement of patients and family caregivers in value assessment is pivotal since they provide valuable contributions to assessment acceptability and relevance. The proposed study aims to use patient-centered techniques and multicriteria decision analysis (MCDA) to evaluate the values of disease-modifying therapies (DMTs) for multiple sclerosis (MS) from the perspectives of patients and family caregivers living in three 'Deep South' States of the US-Alabama, Louisiana, and Mississippi.</p><p><strong>Methods: </strong>This study will follow guidance from the Patient-Centered Outcomes Research Institute (PCORI) for patient engagement and two best practice reports for MCDA from the Professional Society for Health Economics and Outcomes Research (ISPOR) to complete value assessment. Throughout the study, we will engage multiple stakeholders, including patients, family caregivers, healthcare providers, and payers. Forty patients with MS and their family caregivers from Alabama, Louisiana, and Mississippi will be invited to participate in this study. We will intensively train them for value assessment knowledge and MCDA before we engage them in MCDA to determine the value of DMTs for MS.</p><p><strong>Discussions: </strong>Our approach differs from common MCDA since we incorporated a patient-centered framework in this study. Unlike previous studies only briefly inform or prepare participants before the MCDA process, in this study, we will provide basic value assessment trainings for patients and family caregivers to ensure they can effectively engage throughout the patient-centered MCDA process. We expect this study will demonstrate that the patient-centered MCDA approach is feasible and likely leads to improved patients' and family caregivers' engagement in value assessment.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"773-781"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141563959","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Cost Evaluation of COVID-19 Remote Home Monitoring Services in England. 英格兰 COVID-19 远程家庭监控服务成本评估。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-07-01 DOI: 10.1007/s41669-024-00498-3
Sonila M Tomini, Efthalia Massou, Nadia E Crellin, Naomi J Fulop, Theo Georghiou, Lauren Herlitz, Ian Litchfield, Pei Li Ng, Chris Sherlaw-Johnson, Manbinder S Sidhu, Holly Walton, Stephen Morris
{"title":"A Cost Evaluation of COVID-19 Remote Home Monitoring Services in England.","authors":"Sonila M Tomini, Efthalia Massou, Nadia E Crellin, Naomi J Fulop, Theo Georghiou, Lauren Herlitz, Ian Litchfield, Pei Li Ng, Chris Sherlaw-Johnson, Manbinder S Sidhu, Holly Walton, Stephen Morris","doi":"10.1007/s41669-024-00498-3","DOIUrl":"10.1007/s41669-024-00498-3","url":null,"abstract":"<p><strong>Background: </strong>Remote home monitoring services emerged as critical components of health care delivery from NHS England during the COVID-19 pandemic, aiming to provide timely interventions and reduce health care system burden. Two types of service were offered: referral by community health services to home-based care to ensure the right people were admitted to the hospital at the right time (called COVID Oximetry@home, CO@h); and referral by hospital to support patients' transition from hospital to home (called COVID-19 Virtual Ward, CVW). The information collected for the oxygen levels and other symptoms was provided via digital means (technology-enabled) or over the phone (analogue-only submission mode). This study aimed to evaluate the costs of implementing remote home monitoring for COVID-19 patients across 26 sites in England during wave 2 of the pandemic. Understanding the operational and financial implications of these services from the NHS perspective is essential for effective resource allocation and service planning.</p><p><strong>Methods: </strong>We used a bottom-up costing approach at the intervention level to describe the costs of setting up and running the services. Twenty-six implementation sites reported the numbers of patients and staff involved in the service and other resources used. Descriptive statistics and multivariable regression analysis were used to assess cost variations and quantify the relationship between the number of users and costs while adjusting for other service characteristics.</p><p><strong>Results: </strong>The mean cost per patient monitored was lower in the CO@h service compared with the CVW service (£527 vs £599). The mean cost per patient was lower for implementation sites using technology-enabled and analogue data submission modes compared with implementation sites using analogue-only modes for both CO@h (£515 vs £561) and CVW (£584 vs £612) services. The number of patients enrolled in the services and the service type significantly affected the mean cost per patient.</p><p><strong>Conclusions: </strong>Our analysis provides a framework for evaluating the costs of similar services in the future and shows that the implementation of these services benefit from the employment of tech-enabled data submission modes.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"739-753"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362405/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141477168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness of Vedolizumab as a First-Line Advanced Therapy Versus Adalimumab Treatment Sequences for Ulcerative Colitis in Italy. 意大利维多单抗作为溃疡性结肠炎一线晚期疗法与阿达木单抗治疗序列的成本效益对比。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-06-11 DOI: 10.1007/s41669-024-00497-4
Jonathan Salcedo, Daniel Hill-McManus, Chloë Hardern, Oyin Opeifa, Raffaella Viti, Ludovica Siviero, Antonio Saverio Roscini, Gennaro Di Martino
{"title":"Cost-Effectiveness of Vedolizumab as a First-Line Advanced Therapy Versus Adalimumab Treatment Sequences for Ulcerative Colitis in Italy.","authors":"Jonathan Salcedo, Daniel Hill-McManus, Chloë Hardern, Oyin Opeifa, Raffaella Viti, Ludovica Siviero, Antonio Saverio Roscini, Gennaro Di Martino","doi":"10.1007/s41669-024-00497-4","DOIUrl":"10.1007/s41669-024-00497-4","url":null,"abstract":"<p><strong>Background: </strong>Today, there are many treatment options available for the management of ulcerative colitis, creating challenges in selecting the most efficacious and cost-effective treatment sequences. Treatments in the anti-tumor necrosis factor alpha (TNFα) therapeutic class, as well as vedolizumab, are widely used and endorsed as first-line options according to treatment guidelines. The aim of this study was to compare treatment sequences involving vedolizumab and the anti-TNFα treatment adalimumab in terms of cost-effectiveness in the treatment of moderately to severely active ulcerative colitis in Italy.</p><p><strong>Methods: </strong>A cost-effectiveness model comparing treatment sequences within the Italian National Health Service in terms of costs and quality-adjusted life years (QALYs) with a lifetime horizon was developed. The analysis focused on the relative positioning of vedolizumab and adalimumab, leveraging the results of the landmark head-to-head VARSITY clinical trial as key inputs. The robustness of the results was investigated through a range of sensitivity and scenario analyses.</p><p><strong>Results: </strong>The strategy of vedolizumab as a first-line advanced therapy followed by adalimumab was associated with higher costs and health benefits compared with first-line adalimumab followed by vedolizumab. The incremental cost-effectiveness ratio was €16,146/QALY, which was found to be robust to changes to inputs associated with areas of high uncertainty.</p><p><strong>Conclusion: </strong>This economic evaluation estimated a 94% probability that vedolizumab as a first-line advanced therapy is cost-effective at a threshold of €33,004/QALY when compared with first-line adalimumab sequences. Using clinical trial evidence to inform the efficacy of second-line treatments estimated that the effectiveness of anti-TNFα treatments is not substantially reduced by vedolizumab exposure.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"701-714"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362413/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141301202","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Impact of Value-Driven Healthcare Strategies for Biosimilar Adoption: The Singapore Story. 价值驱动型医疗保健战略对采用生物仿制药的影响:新加坡的故事。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-07-23 DOI: 10.1007/s41669-024-00491-w
She Hui Tan, Louise Gek Huang Goh, Benjamin Shao Kiat Ong, Darren Sze Guan Ng, Liang Lin, Raymond Chee Hui Ng, Bernard Yu-Hor Thong, Kwong Ng
{"title":"Impact of Value-Driven Healthcare Strategies for Biosimilar Adoption: The Singapore Story.","authors":"She Hui Tan, Louise Gek Huang Goh, Benjamin Shao Kiat Ong, Darren Sze Guan Ng, Liang Lin, Raymond Chee Hui Ng, Bernard Yu-Hor Thong, Kwong Ng","doi":"10.1007/s41669-024-00491-w","DOIUrl":"10.1007/s41669-024-00491-w","url":null,"abstract":"<p><strong>Background: </strong>Healthcare sustainability is a global challenge. Various value-driven healthcare strategies have been implemented by Singapore's national health technology assessment (HTA) agency, the Agency for Care Effectiveness (ACE). Considering the high and growing expenditure on biologics, strategies have been implemented to drive the use of biosimilars. As Singapore has reached the 5-year mark since the subsidy listing of the first monoclonal antibody biosimilar infliximab, this review aimed to evaluate the impact of these strategies on the changes in adoption rates, utilisation, spending and cost savings for biosimilars in the public healthcare sector.</p><p><strong>Methods: </strong>A retrospective cross-sectional study was conducted using aggregated drug utilisation data from all public healthcare institutions. Five monoclonal antibodies with biosimilars, namely infliximab, adalimumab, trastuzumab, rituximab and bevacizumab, were included in this study. The outcomes evaluated were the monthly trends for utilisation volume, proportion attributed to biosimilar use, and drug spending up to December 2022. The simulated cost savings associated with biosimilar adoption were also reported.</p><p><strong>Results: </strong>After subsidy implementation, an upward trend in biosimilar use and proportion attributed to biosimilar adoption was observed, while spending reduced substantially. The adoption rate of most biosimilars reached more than 95% within 1 year of listing. Drugs with more than one approved biosimilar brand at the time of subsidy listing reported substantial price reductions of over 80%. Overall, spending for the five monoclonal antibodies have significantly reduced after biosimilar subsidy listing, with an estimated cumulative cost savings of $136 million over 5 years.</p><p><strong>Conclusion: </strong>Value-driven healthcare strategies implemented in Singapore's public healthcare institutions have contributed to high adoption rates of biosimilars and have improved affordable access through lower treatment costs. This in turn has led to significant cost savings to the healthcare system.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"679-688"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362392/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141748761","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
NICE's Pathways Pilot: Pursuing Good Decision Making in Difficult Circumstances. NICE 的 Pathways 试点项目:NICE Pathways Pilot: Pursuing Good Decision Making in Difficult Circumances.
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-04-13 DOI: 10.1007/s41669-024-00490-x
Dawn Lee, Darren Burns, Ed Wilson
{"title":"NICE's Pathways Pilot: Pursuing Good Decision Making in Difficult Circumstances.","authors":"Dawn Lee, Darren Burns, Ed Wilson","doi":"10.1007/s41669-024-00490-x","DOIUrl":"10.1007/s41669-024-00490-x","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"645-649"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362421/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140868456","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Psychometric Properties of the EQ-5D-5L in Patients with Alopecia Areata. 脱发患者 EQ-5D-5L 的心理计量特性
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-07-06 DOI: 10.1007/s41669-024-00504-8
Andrew Lloyd, Daniel Aggio, Caleb Dixon, Ernest H Law, Thomas Price
{"title":"Psychometric Properties of the EQ-5D-5L in Patients with Alopecia Areata.","authors":"Andrew Lloyd, Daniel Aggio, Caleb Dixon, Ernest H Law, Thomas Price","doi":"10.1007/s41669-024-00504-8","DOIUrl":"10.1007/s41669-024-00504-8","url":null,"abstract":"<p><strong>Background: </strong>For many decision makers in Health Technology Assessment the EQ-5D-5L is the standard measure of health-related quality of life (HRQL). However, evidence has shown the limitations of the measure in certain disease areas, including dermatology. Alopecia areata (AA) is associated with a significant HRQL impact, partly due to the emotional impact of hair loss.</p><p><strong>Objectives: </strong>This study explores the psychometric properties of the EQ-5D-5L in people with AA in reference to the short-form 36 version 2 (SF-36v2), the Alopecia Areata Patient Priority Outcomes (AAPPO), the Severity of Alopecia Tool (SALT) and the Patient Global Impressions of Change (PGI-C).</p><p><strong>Methods: </strong>Data from participants with AA enrolled in the ALLEGRO-2b/3 trial (NCT03732807) of ritlecitinib were analysed. Participants completed the AAPPO measure (an AA-specific measure assessing emotional symptoms and activity limitations), PGI-C, EQ-5D-5L and SF-36v2 across 48-weeks of follow up. Extent of scalp hair loss was assessed using the SALT. Ceiling effects, known groups validity, convergent validity and responsiveness were examined. Known groups were defined by SALT score and a PGI-C defined response from baseline. Exploratory factor analysis was also performed.</p><p><strong>Results: </strong>Data were available from 612 adult participants. Ceiling effects were observed for the EQ-5D-5L (55.3-61.2%) and analyses suggested that the EQ-5D did not capture important differences between patients that the SF-36v2 did. The EQ-5D-5L very weakly correlated with SALT score, whereas the AAPPO correlated more strongly with the extent of hair loss. Compared with the EQ-5D-5L, the AAPPO was better able to discriminate between known groups defined by SALT and PGI-C. An exploratory factor analysis suggested that the EQ-5D-5L had limitations in content validity compared with the AAPPO.</p><p><strong>Conclusions: </strong>The EQ-5D-5L may not adequately measure the burden of AA on patients' HRQL. Insensitivity to the burden of AA suggests that the EQ-5D-5L may not measure treatment-related benefit with hair regrowth. Data from other measures could be considered if they are shown to be more relevant.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"715-725"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362414/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141538330","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
ENTIMOS: Decision Support Tool Highlights Potential Impact of Non-intravenous Therapies for Multiple Sclerosis on Patient Care via Clinical Scenario Simulation. ENTIMOS:决策支持工具通过临床情景模拟,强调多发性硬化症非静脉注射疗法对患者护理的潜在影响。
IF 2
PharmacoEconomics Open Pub Date : 2024-09-01 Epub Date: 2024-07-11 DOI: 10.1007/s41669-024-00493-8
Richard Nicholas, Erik Scalfaro, Rachel Dorsey, Zuzanna Angehrn, Judit Banhazi, Roisin Brennan, Nicholas Adlard
{"title":"ENTIMOS: Decision Support Tool Highlights Potential Impact of Non-intravenous Therapies for Multiple Sclerosis on Patient Care via Clinical Scenario Simulation.","authors":"Richard Nicholas, Erik Scalfaro, Rachel Dorsey, Zuzanna Angehrn, Judit Banhazi, Roisin Brennan, Nicholas Adlard","doi":"10.1007/s41669-024-00493-8","DOIUrl":"10.1007/s41669-024-00493-8","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Administration of intravenous (IV), high-efficacy treatments (HETs) for the treatment of multiple sclerosis (MS) poses a high resourcing and planning burden on infusion centres, resulting in treatment delays that may increase the risk of breakthrough disease activity. Simulation tools can be used to systematically analyse capacity scenarios and identify and better understand constraints, therefore enabling decision-makers to optimise patient care. We have previously applied ENTIMOS, a discrete event simulation model, to assess scenarios of patient flow and care delivery using real-life data inputs from the neurology infusion suite at Charing Cross Hospital London. The model predicted that, given current capacity and projected demand, patients would experience high-risk treatment delays within 30 months.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;This study aimed to address key healthcare challenges for MS patient care management as seen from a neurologist's perspective. We used ENTIMOS to predict the impact of several distinct and clinically plausible scenarios on patient waiting times at the same MS infusion suite and to quantify mitigation strategies needed to assure continuity of care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We used real-world experience of an expert neurologist to identify five clinical scenarios: (1) switching patients to a subcutaneous (SC) MS treatment of the same therapeutic agent, in the same hospital setting; (2) extending opening times to the weekend; (3) reducing the number of infusion chairs (to simulate social distancing measures applied during the coronavirus disease 2019 [COVID-19] pandemic); (4) increasing demand for infusions; and (5) increasing the scheduling approval time. Patient waiting time for next due infusion and time to high-risk treatment delays (≥ 30 days) were the main analysed model outputs. Previously published base case results were used as reference. All hypothetical scenarios were run over a 3-year horizon (with the exception of scenario 1, which was run over a 3- and 5-year horizon). Strategies to mitigate treatment delays were analysed and discussed.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Switching 50% of patients to SC treatment of the same therapeutic agent administered in hospital postponed the predicted high-risk treatment delays to shortly beyond the 3-year simulation timeframe (month 38). Weekend opening reduced waiting times from 20 days to 4 days and prevented treatment delays, however, at elevated labour costs. Reducing the infusion chairs increased waiting time to 53 days on average and 86 days at the end of the simulation, leading to high-risk treatment delays within 6 months. An increased demand for infusions increased waiting time to 26 days on average and 47 days at the end of the simulation, leading to high-risk treatment delays within 22 months. Prolonged scheduling approval time did not reduce the waiting time, nor postpone the high-risk treatment delays.&lt;/p&gt;&lt;p&gt;&lt;stro","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"755-764"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11362419/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141580453","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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